Novartis’ initial jump into gene therapies with pioneering CAR-T drug Kymriah has run into manufacturing challenges that have slowed expansion in the U.S. But with approval of its second gene therapy, Zolgensma, expected by the end of the month, and more in the pipeline, CEO Vasant Narasimhan is confident that the manufacturing network Novartis is building is up to the task.
The FDA is scheduled to decide by May 31 whether to approve Zolgensma for multiple forms of the genetic disease spinal muscular atrophy (SMA). The company is testing it against the often fatal SMA Type 1, as well as SMA Type 2, a progressive debilitating version of the disease.
In a call Wednesday, David Lennon, who heads the Zolgensma program, told analysts that the company is prepared for a quick launch whatever the FDA approves, Reuters reports.
“Ultimately, they will have to weigh in on exactly where we land on the label,” Lennon said. “We remain very confident around our ability to supply the market under a range of scenarios for what the FDA might grant us in terms of approval.”
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Lennon’s comments were essentially a reiteration of what Narasimhan told investors on an earnings call recently. He said that Novartis has “established readiness ahead of our U.S. approval” with 60 medical centers onboard to send blood for genetic manipulation and then deliver the re-engineered product. The current arrangements will allow Novartis to reach 80% of infants with SMA.
As for manufacturing, Narasimhan said the Swiss drugmaker now has more than 1 million square feet of manufacturing being prepared to ramp up.
“We’re continuing to build supply with the acquisition of a manufacturing site in Colorado,” referring to the recent deal for a former AstraZeneca plant in Longmont, Colorado, which has nearly 700,000 square feet of manufacturing space.
Paul Hudson, who is CEO of Novartis Pharmaceuticals, told investors that even before the company got the Colorado facility, it was ready to meet demand for Zolgensma over the next year or so. With the addition of the new facility, Novartis has “more than enough capacity to deal with what we think could be an unprecedented demand,” he said.
And that is just one piece of the manufacturing puzzle to handle the company’s big move into cell and gene therapies. Narasimhan pointed out that during the earnings call that the company continues to look at using ex vivo cell therapies in a range of other conditions, and “next-generation technologies” to address them.
“That’s part of the reason we built out the global manufacturing base that we have, with a manufacturing center now in the United States, three manufacturing centers in Europe, a manufacturing center in China and a manufacturing center in Japan as well as lentiviral production capability, both in-house and external, that we’re building,” Narasimhan said. “We believe then we would be the logical company to be able to scale ex vivo cell therapies across a range of indications.”
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The company is also making progress with its vanguard gene therapy Kymriah, which is approved for treating certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). It had $45 million in sales last quarter but has been offered mostly to pediatric patients because manufacturing issues have kept some doses from meeting the original specifications.
The company hopes to convince the FDA to change the drug’s label to be more in line with the specifications it has seen in the clinical setting so that it can be provided to more patients. In the meantime, some patients are also getting the drug under the company’s compassionate care protocols. Given the seriousness of the disease, some doctors want to use the drug on patients even when the specs are not exact.
Susanne Schaffert, CEO of Novartis Oncology, says the FDA recently allowed the company to increase manufacturing capacity, and authorities in the EU, Switzerland, Australia, Japan and Canada have approved the drug for commercial specification in line with the clinical reality.
With the approval of Kymriah in Europe, the drugmaker announced it will build a CHF90 million ($91.5 million) plant in Switzerland that eventually may employ up to 450 workers.